ExecutiveChronicles | Drug Repurposing – Finding New Uses for Existing Pharmaceuticals | Drug repurposing can accelerate drug development, overcoming some of the challenges of traditional drug discovery. These include high research and development costs, long timelines for drug approval, low success rates, and revenue loss from patent expiry.
The process reduces the cost and time to reach human clinical trials, particularly in rare diseases. It also helps researchers with limited knowledge of disease mechanisms to test drugs for their efficacy in a new indication.
Cost
Drug repurposing is an approach to finding new medicines for unmet medical needs. It is a cost-effective alternative to traditional drug development that can reduce the time required from years to just months. However, there are several challenges associated with this approach. For example, the lack of access to clinical trial data can delay the progress of drug repurposing efforts. Moreover, repurposing requires a deep understanding of the disease mechanisms and the drug mechanism of action to ensure that the repurposed drug is effective.
Despite these barriers, repurposing has the potential to improve the quality of life for patients with rare diseases and other conditions. It can also accelerate the drug discovery process by cutting research costs. While traditional drug discovery focuses on target identification, repurposing targets disease mechanisms or cell functions. This allows researchers to focus on a narrower group of targets and decreases the risk of developing drugs with severe side effects.
Repurposing has been a successful strategy for pharmaceutical companies, but it is a complex and expensive undertaking. In order to reduce the risk of failure, it is important to invest in early-stage screening and a systematic approach to drug repurposing. In addition, it is important to use knowledge graphs and other tools to organize and analyze complex biological data. Using these tools, scientists can identify direct and indirect relationships between diseases and molecules.
Another barrier to drug repurposing is the difficulty of finding sufficient numbers of patients to conduct a clinical trial. This is especially problematic for rare diseases, where the number of potential patients is small. Several initiatives have been developed to reduce this barrier, including collaborative efforts with academic institutions and NGOs. In addition, the NIH has launched an initiative to support drug repurposing in rare diseases and unmet medical needs.
Many experts claim that repurposing drugs can be cheaper, faster, and less risky than traditional drug development approaches. This is primarily because drug repurposing can bypass the early stages of clinical testing, which establishes the safety of the drug. In addition, repurposing drugs can benefit from the existing infrastructure of the pharmaceutical industry. This includes regulatory agencies, pharmaceutical companies, and public-private partnerships.
Efficacy
Drug repurposing is the process of finding new therapeutic uses for existing drugs. This method is typically faster, less expensive, and more effective than traditional de novo drug development. It is also a critical component of the search for treatments for rare diseases. Currently, there are no licensed treatments for 95% of rare diseases. Repurposing can help reduce the time to market for these drugs, allowing companies to get their products to patients sooner and increase their revenue.
Developing a new drug takes decades and costs hundreds of millions of pounds, but repurposing studies can cost less than half a million pounds and be completed in 2 to 3 years. In addition, repurposed drugs can be tested for efficacy in a shorter period of time. Moreover, repurposed drugs have already passed preclinical and early clinical testing, which means that they are safer to test in humans than new compounds.
However, there are a number of barriers to drug repurposing. Among the most common are inadequate resources and limited subject matter expertise. In addition, repurposing is often a low priority for pharmaceutical companies because of the lack of return on investment. Nevertheless, the industry has a wide range of options for increasing the efficiency and effectiveness of drug repurposing.
One way to improve the efficiency of repurposing is to use a target-centric approach. This approach matches a new disease with an existing drug that has been proven to modulate the target. This method is particularly useful for repurposing drugs for rare diseases. The REMEDi4ALL consortium, for example, is utilizing this approach to develop drugs for EB and other rare diseases.
Another method is to build a knowledge graph, which organizes complex biological data into a network. This helps researchers quickly identify and analyze biological concepts’ direct and indirect relationships. This allows researchers to understand disease biology more quickly, which in turn helps them identify repurposing targets.
The most common facilitators for drug repurposing discussed in the literature were collaborative initiatives, compound libraries and databases, computational-based strategies and tools, and regulatory modifications. However, some critics have questioned the value of drug repurposing and believe that it may divert attention from the pursuit of novel medicines.
Safety
Drug repurposing is a promising strategy to uncover potential new treatments for unmet medical needs. It can reduce costs, shorten timelines, and increase ROI, but its success is dependent on many factors. Hidden skeletons need to be uncovered, and overly optimistic commercial assumptions need to be challenged before a project can be successful.
One of the most significant barriers to drug repurposing is the lack of available resources. Another is the difficulty in accessing and interrogating clinical trial data, especially from abandoned compounds. This is why it is important for repurposing to be part of a multi-partner approach. Collaborations between pharmaceutical companies, academia, and non-profit organizations can help accelerate the process of repurposing drugs.
Repurposing is often based on disease-centric approaches that match diseases with approved or failed drugs that have homologous underlying mechanisms. This approach allows for a faster clinical development process and avoids costly re-inventing the wheel. This is particularly true for rare diseases where repurposing can lead to treatment options that are not otherwise available.
Despite the high cost of bringing a new drug to market, repurposing is gaining momentum as an alternative to traditional drug discovery. It is also a way to address unmet medical needs in underserved regions and boost sales of existing products. In addition, it can save time and money by bypassing the early stages of drug development.
The benefits of repurposing include greater return on investment, extended patent life, and mitigating the risk of failure. Regulatory agencies are increasingly interested in encouraging this approach to drug development. However, it is important to ensure that any repurposed drug has an acceptable safety profile and efficacy in the new indication.
Scientists can use computational tools to systematically identify potential repurposing candidates and assess their safety and toxicity. These software programs can automatically extract and analyze scientific literature, patents, and clinical trials data. They can then evaluate the evidence in a standardized manner and rank candidates for further investigation.
Repurposing can be used to treat a variety of diseases and conditions, from rare and neglected disorders to oncology and infectious diseases. It can even be used to improve existing medications for chronic conditions such as psoriasis.
Regulatory
Drug repurposing can be a cost-effective and time-efficient approach to discovering new treatments for unmet medical needs. However, it can be difficult to overcome regulatory hurdles that hinder its development. These include patents, regulatory pathways, and limited incentives. In addition, repurposing requires large amounts of data from multiple sources, including genomic and proteomic information. This can be difficult to manage, especially when repurposing drugs for rare diseases.
Despite the challenges, there are several ways to address these issues and make drug repurposing more viable. One is to develop a comprehensive strategy that includes all aspects of translational research, including clinical operations and patient engagement. Another is to establish collaborations with different partners in industry, academia, and government. This will ensure that the appropriate expertise is available to support repurposing initiatives. These partnerships should also facilitate the exchange of data and expertise.
In addition, repurposing efforts must be timed correctly to take advantage of patents on original compounds. This is important because drug discovery and design can be a 10-to-17-year process, and it takes an average of 10 years to reach the market. Moreover, the returns on investment for drug repurposing can be much higher than that of traditional drug development.
The first step in a successful repurposing project is to develop a disease model. This is an investigational framework of key biological pathways and networks implicated in a disease that can be used to match existing drugs that modulate these mechanisms. Disease models are built using different types of data, including genomic and proteomic information, cellular interactions, and insights from the scientific literature.
Many pharmaceutical companies focus on developing products for specific categories of diseases or conditions and can abandon projects that do not fit their priorities. For example, AstraZeneca sold its shelved schizophrenia drug candidate to Millendo Therapeutics in 1994, citing that the compound’s hormonal side effects might make it effective against polycystic ovary syndrome (POS).
Another barrier is the lack of resources for repurposing. In a recent survey, respondents identified inadequate financial support and subject matter experts as the most prominent barriers to drug repurposing. Additionally, they mentioned that repurposing can be expensive for all stakeholders, including the public sector, and can distract from other important research activities.
